Systematic Literature Review of the Economic and Quality of Life Burden of Congenital and Immune-Mediated Thrombotic Thrombocytopenic Purpura

These findings underscore the meaningful patient burden observed across the TTP spectrum and highlight the need for further research to evaluate the cost-effectiveness of emerging therapies in cTTP. However, the predominance of iTTP data reflects ongoing challenges in evidence generation for rare TTP subtypes such as cTTP.